Rare Disease Month Developments, Part 1 – The Good: RPD PRV Program Renewed, FDA Rare Disease Hub’s 2026 Strategic Agenda & Plausible Mechanism Draft Guidance On Its Way February 17, 2026 By Mark A. Tobolowsky & James E. Valentine & Frank J. Sasinowski —

https://www.thefdalawblog.com/2026/02/rare-disease-month-developments-part-1-the-good-rpd-prv-program-renewed-fda-rare-disease-hubs-2026-strategic-agenda-plausible-mechanism-draft-guidance-on-its-way/?utm_source=rss&utm_medium=rss&utm_campaign=rare-disease-month-developments-part-1-the-good-rpd-prv-program-renewed-fda-rare-disease-hubs-2026-strategic-agenda-plausible-mechanism-draft-guidance-on-its-way February is Rare Disease Month, designated as such to raise awareness and to support the millions of individuals affected by rare diseases. It is also an extremely meaningful time for those of us at HPM who work regularly with sponsors to help get therapies to patients suffering with rare diseases despite the unique challenges getting these through the FDA approval gauntlet. That’s why we are thrilled to see this month start off with a bang, including the long-awaited renewal of the Rare Pediatric Disease Priority Review Voucher (“RPD PRV”) program, the publication of FDA’s Rare Disease Innovation Hub’s Strategic Agenda for 2026, and a draft guidance on the Plausible Mechanism Pathway under review. At the same time, we have seen recent reminders that such challenges persist, which we will cover in a Part 2 blog post in the coming days.